January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
Phase 1 and Phase 2 Study Results Find Potential Treatment for Severe Cystic Fibrosis
August 5th 2017Vertex Pharmaceuticals recently announced positive data from phase 1 and 2 studies of 3 triple combination regimens in cystic fibrosis patients with one F508del mutation and one minimal function mutation (F508del/Min). These results are the first to present the potential of treating the underlying cause of those with difficult-to-treat cystic fibrosis.
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This Week in Managed Care: August 4, 2017
August 4th 2017This week, the top managed care stories included bipartisan proposals for fixing the Affordable Care Act (ACA); FDA approved a targeted therapy for patients with acute myeloid leukemia who have a rare genetic mutation; and a study found that more patients have gained access to clinical trials after the ACA was implemented.
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ICER Seeks Comments for Proposed Changes for Orphan Drugs Assessments
August 2nd 2017The Institute for Clinical and Economic Review is seeking public comments on its recently released outline of proposed adaptations involving the assessment of certain treatments for ultra-rare conditions, also known as orphan drugs.
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CAR-T Cells Can Increase Clinical Remission Rates in Multiple Myeloma Patients
August 2nd 2017The new immunotherapy of chimeric antigen receptor (CAR)-T cells has demonstrated the ability to increase clinical remission in multiple myeloma patients by targeting the B-cell maturation protein that participates in disease progression.
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Breakthrough Therapy Designation for Venetoclax in AML
July 28th 2017Venetoclax (Venclexta), in combination with low dose cytarabine, has been granted a breakthrough therapy designation for use in elderly, treatment-naive patients with acute myeloid leukemia (AML) who cannot handle intensive chemotherapy.
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Patient Questionnaires Can Predict Clinical Outcomes in Patients With Multiple Sclerosis
July 26th 2017Patient-reported outcomes (PROs) can demonstrate the effects of a disease or treatment. By using patient questionnaires, the results can be analyzed to predict clinical outcomes in medical research for diseases, including multiple sclerosis (MS).
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Mechanism for Checkpoint Inhibitor Resistance Identified in Invasive Bladder Cancer
July 25th 2017Researchers have identified a cellular signaling pathway that when activated by mutations may prevent immune cells from infiltrating bladder tumors, thus making the cancer resistant to immune checkpoint inhibitors.
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Nivolumab, Alone or With Ipilimumab, Met the Disease Control Rate in Malignant Pleural Mesothelioma
July 19th 2017In a late-breaking abstract at the 2017 American Society of Clinical Oncology Annual Meeting, researchers presented an interim analysis of a randomized phase 2 trial of second- or third-line nivolumab, with or without ipilimumab, in patients with second- or third-line malignant pleural mesothelioma.
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Managing heavily pretreated, often less fit, patients with relapsed/refractory multiple myeloma is a challenge in routine practice, as illustrated by the fact progression-free survival remains short, although daratumumab-based combination therapies are proven effective, according to results presented during a poster session at the 2017 American Society of Clinical Oncology Annual Meeting.
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Phase 1 Study Results at ASCO Support First-Line Use of Daratumumab in Multiple Myeloma
July 17th 2017A phase 1 study, presented at the 2017 Annual Meeting of the American Society of Clinical Oncology, found that using daratumumab, an antibody that binds and inhibits the CD38 receptor, can improve patient response to treatment.
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Updated trial results at the 2017 Annual Meeting of the American Society of Clinical Oncology showed the combinations of daratumumab with either lenalidomide and dexamethasone or bortezomib and dexamethasone prolonged progression-free survival for patients with relapsed refractory multiple myeloma.
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Investor Reactions to Orphan Drug Designation Strongest for Oncology Therapies, Study Finds
July 14th 2017A new study examines the impact that a pharmaceutical’s designation as an orphan drug can have on its manufacturer’s stock prices, and finds that the largest returns were for oncology drugs.
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FDA Grants Full Approval, Expands Indications for Blinatumomab in ALL
July 12th 2017Blinatumomab is now fully approved as a treatment for relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), following FDA assessment of new trial data on survival outcomes and its use in certain clinical subgroups.
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Genome Sequencing Reveals Rare Disease Risk in 1 of 5 Generally Healthy Adults
July 12th 2017An analysis of whole-genome sequencing (WGS) in healthy adults in the primary care setting found that 1 in 5 had a previously unrecognized variant associated with a rare disease, according to a study published in Annals of Internal Medicine.
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Novartis' CAR-T Therapy for Pediatric Leukemia to Face FDA Advisory Panel
July 11th 2017Novartis’ chimeric antigen receptor T-cell (CAR-T) therapy for treating pediatric leukemia is on the cusp of being the first FDA-approved gene therapy, which will lead to new developments and utilizations of CAR-T therapy for treating other advanced blood cancers.
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New Antibody Demonstrates 2-Pronged Approach in Treating Blood Cancer
July 9th 2017A study published by BloodAdvances, a Journal of the American Society of Hematology, demonstrated the potential of the 2-pronged approach in treating non-Hodgkin lymphoma, multiple myeloma, and acute myeloid leukemia.
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Rare Disease Can Be Misdiagnosed as Rheumatoid Arthritis
July 3rd 2017Whipple’s disease (WD) is a rare disease that predominantly affects middle-aged white men, and its diagnosis is often delayed because it is misdiagnosed as rheumatoid arthritis. A new study in Orphanet Journal of Rare Diseases aimed to describe clinical symptoms of WD and appropriate treatment.
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Novel Antibody May Restore Immune Response to Rituximab for CLL
June 29th 2017Among a group of patients with chronic lymphocytic leukemia (CLL) who were previously unresponsive to rituximab, a new antibody could help enable the immune system to attack the cancer cells, according to a study in PLOS One.
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Nivolumab Improved Patient-Reported QOL Compared With Investigator's Choice: CheckMate 141
June 28th 2017The study, published in Lancet Oncology, has found that nivolumab reduced the rate of clinically meaningful deterioration compared with investigator’s choice among platinum-refractory patients being treated for recurrent or metastatic head and neck cancer.
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Infusing a Sense of Urgency in Cancer Care: The Biden Cancer Initiative
June 27th 2017On Monday, June 26, 2017, the Biden Foundation announced the launch of the Biden Cancer Initiative, their new venture to continue the fight to make progress in cancer prevention, detection, treatment, and care.
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